Capricor’s HOPE-3 Phase 3 Cardiac Results for Deramiocel Win American Heart Association Outstanding Research Award in Pediatric Cardiology
The overall cardiac endpoint missed statistical significance, while subgroup and scar-imaging analyses favored Deramiocel.
Sentiment and the balance of points
Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.
Rhea-AI Summary
Capricor Therapeutics (CAPR) presented new HOPE-3 cardiac data for Deramiocel, with the abstract receiving an American Heart Association pediatric cardiology research award.
The 106-patient, 12-month Duchenne muscular dystrophy trial met its primary upper-limb function endpoint. The key secondary endpoint, heart pumping function measured by left ventricular ejection fraction, favored Deramiocel by 1.8 percentage points versus placebo but missed statistical significance (p=0.09). The prespecified cardiomyopathy subgroup showed a 2.8-point advantage; scar imaging showed 3.0 fewer newly scarred heart segments. A post hoc, non-randomized natural history comparison also favored Deramiocel. Subgroup, scar-imaging and natural history p-values were nominal, without adjustment for multiple comparisons. The biologics license application remains under FDA review, with a November 22, 2026 target action date.
How this balance works
Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.
It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.
Rhea-AI Sentiment measures something else, the tone of the wording.
Positive
- Major pointHOPE-3 met its primary upper-limb function endpoint in the 12-month randomized trial.
- Minor pointPrespecified cardiomyopathy subgroup: heart pumping function favored Deramiocel by 2.8 percentage points versus placebo (nominal p=0.02).
- Minor pointHeart scarring increased by 3.0 fewer new segments versus placebo (nominal p=0.02).
- Minor pointNatural history comparison: overall heart pumping function favored Deramiocel by 2.3 percentage points (nominal p=0.02).
- Minor pointNatural history cardiomyopathy comparison: heart pumping function favored Deramiocel by 2.8 percentage points (nominal p=0.01).
- Minor pointNatural history scar comparison showed 2.9 fewer new scarred segments with Deramiocel (nominal p=0.01).
Negative
- Moderate pointKey secondary cardiac endpoint missed statistical significance: +1.8 percentage points versus placebo, p=0.09.
- Moderate pointDeramiocel's application remains under FDA review, with a November 22, 2026 target action date.
- Minor pointSubgroup, scar-imaging and natural history p-values were not adjusted for multiple comparisons.
- Minor pointNatural history comparisons were post hoc and non-randomized.
- Minor pointScar-imaging comparisons included only 9 Deramiocel patients, versus 13 placebo patients or 62 natural history patients.
Key Figures
- Patients randomized
- 106 patients
- HOPE-3 Phase 3 trial
- Treatment period
- 12 months
- HOPE-3 randomized trial
- LVEF change, all patients
- +1.8 percentage points (p=0.09)
- Deramiocel vs. placebo; did not reach statistical significance
- LVEF change, cardiomyopathy subgroup
- +2.8 percentage points (p=0.02)
- Deramiocel vs. placebo; subgroup p-value nominal
- Fewer new scarred heart segments
- 3.0 fewer segments (p=0.02)
- Deramiocel vs. placebo; LGE result, p-value nominal
- PDUFA target action date
- November 22, 2026
- Deramiocel BLA remains under FDA review
Key Terms
myocardial fibrosis medical
late gadolinium enhancement medical
left ventricular ejection fraction medical
biologics license application regulatory
pdufa regulatory
AI-generated analysis. How Rhea-AI works. Not financial advice.
SAN DIEGO, Nov. 09, 2026 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the abstract presenting cardiac results from the Company’s HOPE-3 Phase 3 trial of Deramiocel in Duchenne muscular dystrophy (DMD) received the Outstanding Research Award in Pediatric Cardiology at the American Heart Association (AHA) Scientific Sessions 2026 on Saturday, November 7, in Chicago. The presentation included new cardiac MRI data on myocardial fibrosis, measured by late gadolinium enhancement (LGE), and a comparison with matched patients not treated with Deramiocel from a natural history dataset.
The HOPE-3 trial randomized 106 patients with DMD to Deramiocel or placebo for 12 months. The trial met its primary endpoint of upper limb function. The key secondary endpoint was change in left ventricular ejection fraction (LVEF) in the intent-to-treat population. LVEF, the percentage of blood the heart pumps out with each beat, is a standard measure of cardiac function.
Key results include:
| Cardiac measure | Deramiocel vs. placebo HOPE-3, randomized | Deramiocel vs. matched patients Natural history comparison, non-randomized |
| Heart function (LVEF) All patients | +1.8 percentage points p=0.09 | +2.3 percentage points p=0.02 |
| Heart function (LVEF) Patients with cardiomyopathy (prespecified subgroup) | +2.8 percentage points p=0.02 | +2.8 percentage points p=0.01 |
| Heart scarring (LGE) Increase in number of heart segments with scar | 3.0 fewer new segments p=0.02 | 2.9 fewer new segments p=0.01 |
A smaller increase in scarred segments means slower progression of scarring. LVEF: left ventricular ejection fraction. LGE: late gadolinium enhancement, cardiac MRI scar imaging. The natural history comparison, using data from the Duchenne Muscular Dystrophy Cardiac Care Consortium (DMDCCC), is post hoc and non-randomized. The key secondary endpoint (LVEF, all patients) did not reach statistical significance (p=0.09). P-values for the cardiomyopathy subgroup, LGE and the natural history comparison are nominal (not adjusted for multiple comparisons). Number of patients (Deramiocel vs. comparator) — placebo comparison: LVEF, all patients, 39 vs. 44; LVEF, cardiomyopathy subgroup, 32 vs. 32; LGE, 9 vs. 13. Natural history comparison: LVEF, all patients, 39 vs. 75; LVEF, cardiomyopathy subgroup, 32 vs. 60; LGE, 9 vs. 62.
“Cardiomyopathy is the leading cause of death in patients with Duchenne muscular dystrophy. There is no therapy approved specifically to treat DMD cardiomyopathy; therefore, development of novel therapeutics that can slow or stop its progression is of the utmost importance,” said Jonathan Soslow, M.D., MSCI, Professor of Pediatrics (Cardiology) at Vanderbilt University Medical Center and lead author of the abstract. “While the study suggests benefit across all treated patients, the most pronounced improvements in ventricular function were seen in the cardiomyopathy subgroup. Patients on therapy also showed slower progression of scar aggregation in their hearts. As cardiologists, we watch the scar increase and the heart function decrease year after year in patients with Duchenne. Seeing signals of stabilization for both is encouraging, and it is the combination we would want from a cardiac therapy in Duchenne.”
“The one-year HOPE-3 results were published in The Lancet, and these cardiac findings have now received AHA’s Outstanding Research Award in Pediatric Cardiology – independent recognition of the strength of these data. Protecting the heart remains one of the most urgent unmet needs in Duchenne, and we believe Deramiocel could be an important tool in addressing it. We congratulate Dr. Soslow on this recognition and thank him for sharing these results with the cardiology community,” added Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “Our Biologics License Application remains under FDA review, with a PDUFA target action date of November 22, 2026. We look forward to the completion of that review and to the FDA’s continued focus on rare diseases such as Duchenne.”
Presentation Details
Title: Cardiac Effects of Deramiocel in Duchenne Muscular Dystrophy: Randomized Placebo-Controlled Phase-3 Trial Results and Natural History Comparison
Lead Author: Jonathan Soslow, M.D., MSCI, Vanderbilt University Medical Center
Session: Outstanding Research Award in Pediatric Cardiology
Date: November 7, 2026
AHA Scientific Sessions is the American Heart Association’s annual meeting covering the full spectrum of cardiovascular science. Meeting details, including the full program, are available on the AHA Scientific Sessions 2026 website. The presentation slides will be made available in the publications section of the Capricor website.
About the HOPE-3 Study
HOPE-3 is a Phase 3, randomized, double-blind, placebo-controlled trial evaluating Deramiocel in patients with Duchenne muscular dystrophy. The study enrolled 106 patients, randomized to receive Deramiocel or placebo administered intravenously every three months over a 12-month treatment period. One-year results were published in The Lancet in July 2026. Patients who completed the randomized portion of the study were eligible to continue receiving Deramiocel in an open-label extension (OLE).
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, the leading cause of death in DMD. There is no cure, and treatment options remain limited.
About Deramiocel
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert immunomodulatory and anti-fibrotic actions in the preservation of skeletal and cardiac muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. For the treatment of DMD, Deramiocel holds Orphan Drug, RMAT and Rare Pediatric Disease designations in the U.S., and Orphan Drug and ATMP designations in Europe. The Rare Pediatric Disease designation may qualify Capricor for a Priority Review Voucher upon approval.
About Capricor Therapeutics
Capricor Therapeutics (NASDAQ: CAPR) is a biotechnology company dedicated to advancing cell and exosome-based therapeutics for the treatment of rare diseases. Our lead product candidate, Deramiocel, is an allogeneic cardiac-derived cell therapy in late-stage development for the treatment of Duchenne muscular dystrophy (DMD), evaluated in clinical studies for its potential to preserve skeletal and cardiac muscle function. Capricor is also advancing its proprietary StealthX™ exosome platform for the targeted delivery of oligonucleotides, proteins, and small-molecule therapeutics across a range of diseases. At Capricor, we are committed to delivering new therapies for patients with rare diseases. For more information, visit capricor.com and follow Capricor on Facebook, Instagram and X.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including future interactions with regulatory authorities and the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; the potential that required regulatory inspections may be delayed or not be successful, which would delay or prevent product approval; revenue and reimbursement estimates; projected terms of definitive agreements, our financial position, our possible uses of existing cash and investment resources; results of securities litigation; and statements regarding our litigation with Nippon Shinyaku Co., Ltd. and NS Pharma, Inc., including the nature of the dispute, our expectations regarding any legal proceedings, and our ability to commercialize Deramiocel independent of our existing distribution agreement and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission on March 17, 2026 and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as filed with the Securities and Exchange Commission on August 14, 2026. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.
Deramiocel and Capricor’s StealthX™ exosome therapeutics are investigational and have not been approved for commercial use in any indication.
For more information, please contact:
Capricor Media Contact:
Caitlin Kasunich / Raquel Cona
KCSA Strategic Communications
ckasunich@kcsa.com / rcona@kcsa.com
212.896.1241 / 516.779.2630
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
Did Capricor's HOPE-3 trial meet its cardiac endpoint?
The key secondary endpoint, change in left ventricular ejection fraction in all patients, did not reach statistical significance. Deramiocel showed a 1.8-percentage-point advantage versus placebo, with p=0.09. The trial did meet its primary endpoint of upper-limb function.
When is the FDA target action date for Capricor's Deramiocel application?
The FDA target action date for Deramiocel's biologics license application is November 22, 2026. The application remains under review.
How was Deramiocel administered in Capricor's HOPE-3 trial?
HOPE-3 randomized patients to receive Deramiocel or placebo intravenously every three months over a 12-month treatment period. The trial was double-blind, and patients completing the randomized portion were eligible to continue receiving Deramiocel in an open-label extension.
How many patients contributed to Capricor's HOPE-3 cardiac analyses?
The placebo-controlled heart pumping function analysis included 39 Deramiocel patients and 44 placebo patients; its cardiomyopathy subgroup included 32 versus 32. Scar imaging included 9 versus 13. Natural history comparisons used the same Deramiocel counts against 75, 60 and 62 matched patients, respectively, using Duchenne Muscular Dystrophy Cardiac Care Consortium data.